AAV-mediated gene therapy for Sialidosis
Diantha van de Vlekkert, Huimin Hu, Jason A. Weesner…·Alessandra d’Azzo
This study tests AAV-mediated gene therapy for sialidosis in a mouse model, showing restored NEU1 activity and reduced tissue damage.
Author
St. Jude Children's Research HospitalORCID 0000-0001-9980-9042Unclaimed ORCID profile · follow to get notified of new preprints
3 papers
Recent·Most cited
Diantha van de Vlekkert, Huimin Hu, Jason A. Weesner…·Alessandra d’Azzo
This study tests AAV-mediated gene therapy for sialidosis in a mouse model, showing restored NEU1 activity and reduced tissue damage.
Leigh E. Fremuth, Huimin Hu, Diantha van de Vlekkert…·Alessandra d’Azzo
This study shows that Neu1 regulates microglial function by modulating Trem2 sialylation, affecting phagocytosis and cytokine production.
Jason A. Weesner, Ida Annunziata, Diantha van de Vlekkert…·Alessandra d’Azzo
This study shows that altered GM1 catabolism disrupts ER-PM junctions, increases Ca2+ signaling, and promotes synaptic spine formation in a neurodegenerative disease model.